JP2014054250A5 - - Google Patents
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- JP2014054250A5 JP2014054250A5 JP2013184193A JP2013184193A JP2014054250A5 JP 2014054250 A5 JP2014054250 A5 JP 2014054250A5 JP 2013184193 A JP2013184193 A JP 2013184193A JP 2013184193 A JP2013184193 A JP 2013184193A JP 2014054250 A5 JP2014054250 A5 JP 2014054250A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligomer
- group
- oligomer according
- bond
- antisense
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 230000000692 anti-sense effect Effects 0.000 claims 11
- 108091034117 Oligonucleotide Proteins 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 2
- 108010058514 Phosphate-Binding Proteins Proteins 0.000 claims 2
- 102000006335 Phosphate-Binding Proteins Human genes 0.000 claims 2
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000002947 alkylene group Chemical group 0.000 claims 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- UXTZGKSGGBHJOE-UHFFFAOYSA-N CN(C)P(N(C)CC(N)=O)(OC)=O Chemical compound CN(C)P(N(C)CC(N)=O)(OC)=O UXTZGKSGGBHJOE-UHFFFAOYSA-N 0.000 description 1
- UCLQCQZSVIFYKB-UHFFFAOYSA-N CN(C)P(N(CC1)CCN1C(OCCOCCOCCO)=O)(OC)=O Chemical compound CN(C)P(N(CC1)CCN1C(OCCOCCOCCO)=O)(OC)=O UCLQCQZSVIFYKB-UHFFFAOYSA-N 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2013184193A JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2010196032 | 2010-09-01 | ||
| JP2010196032 | 2010-09-01 | ||
| JP2013184193A JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012531987A Division JP5363655B2 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015256962A Division JP6193343B2 (ja) | 2010-09-01 | 2015-12-28 | アンチセンス核酸 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014054250A JP2014054250A (ja) | 2014-03-27 |
| JP2014054250A5 true JP2014054250A5 (fr) | 2014-06-05 |
| JP6141728B2 JP6141728B2 (ja) | 2017-06-07 |
Family
ID=45773054
Family Applications (12)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012531987A Active JP5363655B2 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
| JP2013184193A Active JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
| JP2015256962A Active JP6193343B2 (ja) | 2010-09-01 | 2015-12-28 | アンチセンス核酸 |
| JP2017154320A Active JP6465932B2 (ja) | 2010-09-01 | 2017-08-09 | アンチセンス核酸 |
| JP2019001091A Active JP6647430B2 (ja) | 2010-09-01 | 2019-01-08 | アンチセンス核酸 |
| JP2020003338A Active JP6867619B2 (ja) | 2010-09-01 | 2020-01-14 | アンチセンス核酸 |
| JP2021008202A Active JP6867636B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008200A Active JP6867620B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008201A Active JP6867621B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021058526A Pending JP2021104037A (ja) | 2010-09-01 | 2021-03-30 | アンチセンス核酸 |
| JP2022209270A Pending JP2023036865A (ja) | 2010-09-01 | 2022-12-27 | アンチセンス核酸 |
| JP2024145055A Pending JP2024170458A (ja) | 2010-09-01 | 2024-08-27 | アンチセンス核酸 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012531987A Active JP5363655B2 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
Family Applications After (10)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015256962A Active JP6193343B2 (ja) | 2010-09-01 | 2015-12-28 | アンチセンス核酸 |
| JP2017154320A Active JP6465932B2 (ja) | 2010-09-01 | 2017-08-09 | アンチセンス核酸 |
| JP2019001091A Active JP6647430B2 (ja) | 2010-09-01 | 2019-01-08 | アンチセンス核酸 |
| JP2020003338A Active JP6867619B2 (ja) | 2010-09-01 | 2020-01-14 | アンチセンス核酸 |
| JP2021008202A Active JP6867636B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008200A Active JP6867620B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008201A Active JP6867621B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021058526A Pending JP2021104037A (ja) | 2010-09-01 | 2021-03-30 | アンチセンス核酸 |
| JP2022209270A Pending JP2023036865A (ja) | 2010-09-01 | 2022-12-27 | アンチセンス核酸 |
| JP2024145055A Pending JP2024170458A (ja) | 2010-09-01 | 2024-08-27 | アンチセンス核酸 |
Country Status (20)
| Country | Link |
|---|---|
| US (16) | US9079934B2 (fr) |
| EP (6) | EP4403632A3 (fr) |
| JP (12) | JP5363655B2 (fr) |
| KR (1) | KR101310569B1 (fr) |
| CN (1) | CN103154245B (fr) |
| AU (1) | AU2011296882B2 (fr) |
| CA (1) | CA2809637C (fr) |
| CY (2) | CY1117367T1 (fr) |
| DK (2) | DK2612917T3 (fr) |
| ES (2) | ES2567411T3 (fr) |
| HR (2) | HRP20160336T1 (fr) |
| HU (2) | HUE027321T2 (fr) |
| LT (1) | LT3018211T (fr) |
| PL (2) | PL2612917T3 (fr) |
| PT (1) | PT3018211T (fr) |
| RS (2) | RS59361B1 (fr) |
| SI (2) | SI3018211T1 (fr) |
| SM (2) | SMT201900559T1 (fr) |
| TW (1) | TWI541024B (fr) |
| WO (1) | WO2012029986A1 (fr) |
Families Citing this family (73)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1855694B1 (fr) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Composition antisens permettant de traiter une atrophie musculaire |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| EP2119783A1 (fr) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| SMT201900159T1 (it) | 2008-10-24 | 2019-05-10 | Sarepta Therapeutics Inc | Composizioni di salto di esone per dmd |
| KR20230137491A (ko) | 2009-11-12 | 2023-10-04 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| BR112014018427B1 (pt) * | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
| EP2935584A1 (fr) * | 2012-12-20 | 2015-10-28 | Sarepta Therapeutics, Inc. | Compositions améliorées induisant un saut d'exon pour le traitement d'une dystrophie musculaire |
| KR20240094032A (ko) | 2013-03-14 | 2024-06-24 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| NZ631245A (en) * | 2013-03-14 | 2017-09-29 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| HK1220154A1 (zh) | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
| SG11201607095RA (en) * | 2014-03-12 | 2016-10-28 | Nippon Shinyaku Co Ltd | Antisense nucleic acids |
| US10174319B2 (en) | 2014-05-19 | 2019-01-08 | Knc Laboratories Co., Ltd. | Nucleic acid drug for inducing skipping of variant exon of CD44 gene and increasing expression of normal type CD44 mRNA |
| CN111575282B (zh) * | 2014-06-17 | 2024-05-14 | 日本新药株式会社 | 反义核酸 |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| CN108026531B (zh) * | 2015-09-15 | 2021-09-14 | 日本新药株式会社 | 反义核酸 |
| JP6875684B2 (ja) * | 2015-09-16 | 2021-05-26 | 日本新薬株式会社 | 筋萎縮症治療用アンチセンス核酸 |
| NZ781029A (en) | 2015-10-09 | 2025-07-25 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| EP3359668A4 (fr) | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| KR101686379B1 (ko) | 2016-02-05 | 2016-12-13 | 박정열 | 휴대용 구이기 |
| WO2017205879A2 (fr) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| WO2017205496A1 (fr) | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Procédés de préparation d'oligomères |
| MA45183A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| DK3464306T3 (da) | 2016-05-24 | 2024-05-21 | Sarepta Therapeutics Inc | Fremgangsmåder til fremstilling af phosphorodiamidat-morpholino-oligomerer |
| KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| KR102824513B1 (ko) * | 2016-07-05 | 2025-06-25 | 바이오마린 테크놀로지스 비.브이. | 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드 |
| KR101715572B1 (ko) | 2016-10-11 | 2017-03-22 | 박종찬 | 구이대 승강구조를 갖는 양방향 구이기 |
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| SG10202012839TA (en) | 2016-12-19 | 2021-01-28 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| MD3554554T2 (ro) | 2016-12-19 | 2022-12-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
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| CA3073736A1 (fr) * | 2017-08-31 | 2019-03-07 | Sarepta Therapeutics, Inc. | Methodes permettant de traiter la dystrophie musculaire |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536057A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| EP3784248A4 (fr) * | 2018-04-26 | 2022-08-10 | Sarepta Therapeutics, Inc. | Oligomères induisant le saut d'exon et conjugués d'oligomères pour la dystrophie musculaire |
| AU2019265904A1 (en) * | 2018-05-11 | 2020-11-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| MY209148A (en) * | 2018-06-26 | 2025-06-24 | Nippon Shinyaku Co Ltd | Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
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| WO2022059754A1 (fr) * | 2020-09-18 | 2022-03-24 | 天野エンザイム株式会社 | Procédé de fabrication de lait de pois chiches traité |
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| EP4486386A1 (fr) | 2022-03-03 | 2025-01-08 | Yale University | Compositions et procédés d'administration de polynucléotides thérapeutiques pour saut d'exon |
| JP2025079347A (ja) * | 2022-03-11 | 2025-05-22 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
| EP4493693A1 (fr) | 2022-03-17 | 2025-01-22 | Sarepta Therapeutics, Inc. | Conjugués d'oligomère morpholino phosphorodiamidate |
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